内分泌科

订阅

MedSci主页 / 所有栏目 / 内分泌科

FDA批准了ABO-202基因疗法治疗CLN1的研究性新药申请

2019-05-22 不详 网络

ABO-202基因疗法FDAIND
关注

Abeona是一家专注于基因和细胞疗法的生物医药公司，近日宣布已开始进行1/2期临床试验，以评估其新型基因疗法ABO-202治疗CLN1疾病的有效性。

Abeona是一家专注于基因和细胞疗法的生物医药公司，近日宣布已开始进行1/2期临床试验，以评估其新型基因疗法ABO-202治疗CLN1疾病的有效性。同时，FDA已批准了ABO-202基因疗法治疗CLN1的研究性新药申请（IND）。ABO-202通过AAV9载体，通过静脉内和鞘内递送，将PPT1基因的功能性拷贝递送至中枢神经系统和外周器官的病变细胞。

Abeona总裁兼首席科学官Timothy J. Miller博士说：“在临床前研究中，ABO-202能够有效地将PPT1基因的功能性拷贝递送至中枢神经系统和外周器官，ABO-202能够使CLN1动物模型的存活正常化并介导神经功能的改善。同时，静脉内和鞘内组合给药比单独的给药途径增加有效性，因此可以增强该基因疗法的治疗潜力”。


原始出处：

http://www.firstwordpharma.com/node/1642385#axzz5oeL0ziZx

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

版权声明：
本网站所有内容来源注明为“梅斯医学”或“MedSci原创”的文字、图片和音视频资料，版权均属于梅斯医学所有。非经授权，任何媒体、网站或个人不得转载，授权转载时须注明来源为“梅斯医学”。其它来源的文章系转载文章，或“梅斯号”自媒体发布的文章，仅系出于传递更多信息之目的，本站仅负责审核内容合规，其内容不代表本站立场，本站不负责内容的准确性和版权。如果存在侵权、或不希望被转载的媒体或个人可与我们联系，我们将立即进行删除处理。
在此留言
评论区 (5)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1744187, encodeId=4c771e441879d, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Sep 22 03:33:00 CST 2019, time=2019-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839690, encodeId=106b1839690d6, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Aug 01 17:33:00 CST 2019, time=2019-08-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366839, encodeId=daa536683941, content=学习了，谢谢分享。, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib06iaV3D9Tg5Kzuf9u71gZhPYMiajRtENwicoAABeQtfXOlic8ibhYSy6DvJZWUDVtjRvTfqBffr1XJ6JLtFB5kHicthl/0, createdBy=d41c2055892, createdName=184****9840, createdTime=Mon May 27 00:10:22 CST 2019, time=2019-05-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598027, encodeId=bff9159802e3c, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Fri May 24 10:33:00 CST 2019, time=2019-05-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366612, encodeId=59d936661247, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=71, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed May 22 21:28:07 CST 2019, time=2019-05-22, status=1, ipAttribution=)]
    2019-09-22 wangbingxhy

    #新药申请#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1744187, encodeId=4c771e441879d, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Sep 22 03:33:00 CST 2019, time=2019-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839690, encodeId=106b1839690d6, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Aug 01 17:33:00 CST 2019, time=2019-08-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366839, encodeId=daa536683941, content=学习了，谢谢分享。, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib06iaV3D9Tg5Kzuf9u71gZhPYMiajRtENwicoAABeQtfXOlic8ibhYSy6DvJZWUDVtjRvTfqBffr1XJ6JLtFB5kHicthl/0, createdBy=d41c2055892, createdName=184****9840, createdTime=Mon May 27 00:10:22 CST 2019, time=2019-05-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598027, encodeId=bff9159802e3c, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Fri May 24 10:33:00 CST 2019, time=2019-05-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366612, encodeId=59d936661247, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=71, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed May 22 21:28:07 CST 2019, time=2019-05-22, status=1, ipAttribution=)]
    2019-08-01 智者为医08

    #FDA批准#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1744187, encodeId=4c771e441879d, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Sep 22 03:33:00 CST 2019, time=2019-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839690, encodeId=106b1839690d6, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Aug 01 17:33:00 CST 2019, time=2019-08-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366839, encodeId=daa536683941, content=学习了，谢谢分享。, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib06iaV3D9Tg5Kzuf9u71gZhPYMiajRtENwicoAABeQtfXOlic8ibhYSy6DvJZWUDVtjRvTfqBffr1XJ6JLtFB5kHicthl/0, createdBy=d41c2055892, createdName=184****9840, createdTime=Mon May 27 00:10:22 CST 2019, time=2019-05-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598027, encodeId=bff9159802e3c, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Fri May 24 10:33:00 CST 2019, time=2019-05-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366612, encodeId=59d936661247, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=71, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed May 22 21:28:07 CST 2019, time=2019-05-22, status=1, ipAttribution=)]
    2019-05-27 184****9840

    学习了，谢谢分享。

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1744187, encodeId=4c771e441879d, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Sep 22 03:33:00 CST 2019, time=2019-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839690, encodeId=106b1839690d6, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Aug 01 17:33:00 CST 2019, time=2019-08-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366839, encodeId=daa536683941, content=学习了，谢谢分享。, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib06iaV3D9Tg5Kzuf9u71gZhPYMiajRtENwicoAABeQtfXOlic8ibhYSy6DvJZWUDVtjRvTfqBffr1XJ6JLtFB5kHicthl/0, createdBy=d41c2055892, createdName=184****9840, createdTime=Mon May 27 00:10:22 CST 2019, time=2019-05-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598027, encodeId=bff9159802e3c, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Fri May 24 10:33:00 CST 2019, time=2019-05-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366612, encodeId=59d936661247, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=71, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed May 22 21:28:07 CST 2019, time=2019-05-22, status=1, ipAttribution=)]
    2019-05-24 闆锋旦

    #ABO#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1744187, encodeId=4c771e441879d, content=<a href='/topic/show?id=657a58646ca' target=_blank style='color:#2F92EE;'>#新药申请#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=58646, encryptionId=657a58646ca, topicName=新药申请)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ab3c35313981, createdName=wangbingxhy, createdTime=Sun Sep 22 03:33:00 CST 2019, time=2019-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839690, encodeId=106b1839690d6, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Thu Aug 01 17:33:00 CST 2019, time=2019-08-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366839, encodeId=daa536683941, content=学习了，谢谢分享。, beContent=null, objectType=article, channel=null, level=null, likeNumber=78, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib06iaV3D9Tg5Kzuf9u71gZhPYMiajRtENwicoAABeQtfXOlic8ibhYSy6DvJZWUDVtjRvTfqBffr1XJ6JLtFB5kHicthl/0, createdBy=d41c2055892, createdName=184****9840, createdTime=Mon May 27 00:10:22 CST 2019, time=2019-05-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598027, encodeId=bff9159802e3c, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Fri May 24 10:33:00 CST 2019, time=2019-05-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=366612, encodeId=59d936661247, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=71, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed May 22 21:28:07 CST 2019, time=2019-05-22, status=1, ipAttribution=)]
    2019-05-22 坚强007

    向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬！

    0